Details of the Drug

General Information of Drug (ID: DMH6GLU)

Drug Name

TAK-754

Synonyms

BAX 888

Indication

Disease Entry	ICD 11	Status	REF
Haemophilia A	3B10.0	Phase 1/2	[1]
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Drug Type

Gene therapy

Cross-matching ID

Molecular Interaction Atlas of This Drug

Drug Therapeutic Target (DTT)

Molecular Interaction Atlas (MIA)

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Molecular Expression Atlas of This Drug

The Studied Disease

Haemophilia A

ICD Disease Classification

3B10.0

Molecule Name	Molecule Type	Gene Name	p-value	Fold-Change	Z-score
Coagulation factor VIII (F8)	DTT	F8	1.08E-22	0.89	1.82

Molecular Expression Atlas (MEA)

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References

1	ClinicalTrials.gov (NCT03370172) A Global, Open-Label, Multicenter, Phase 1/2 Study of the Safety and Dose Escalation of BAX 888, an Adeno-Associated Virus Serotype 8 (AAV8) Vector Expressing B-Domain Deleted Factor VIII (BDD-FVIII) in Severe Hemophilia A Subjects Administered a Single Intravenous Infusion. U.S.National Institutes of Health.
2	A reprieve from hemophilia A, but for how long? Nat Biotechnol. 2020 Oct;38(10):1107-1109.